Hemophilia

Diabetes and HIV May Increase Risk of Hypertension in People with Hemophilia

September 21, 2022

Findings suggest that screening people with hemophilia for diabetes and hypertension might have benefits.

Gene Therapy for Severe Hemophilia B Could Be More Cost Effective Than Current Treatments

September 19, 2022

An analysis found that a single dose of gene therapy would be more cost-effective than on-demand or prophylaxis factor replacement in 92% of cases.

Gene Therapies for Hemophilia. Possibly Curative, Likely Ultraexpensive

September 08, 2022

Three gene therapies in late-stage development are designed to supply working copies of genes that people with hemophilia are missing. They may be one-time, curative treatments, but the seven-figure price tags will be an issue.

Anxiety, Depression, and Diabetes Higher in Older Males with Hemophilia; Cardiovascular Disease Lower

August 19, 2022

Lack of blood clotting may afford some protection against common cardiovascular disease events, such as ischemic stroke.

Gene Therapy for Hemophilia B Shows Promise

August 01, 2022

A small study finds that an adeno-associated virus gene therapy eliminated both spontaneous bleeding and the need for factor IX prophylaxis in patients with hemophilia B.

ICER Draft Report Finds Beti-Cel is Cost-Effective for Blood Disorder

April 13, 2022

ICER said Bluebird Bio could use an outcome-based pricing plan of five yearly payments totaling $2.1 million for patients treated with beti-cel who no longer need blood transfusions.

Study: Thrombotic Events Increase Mortality Risk for People With Polycythemia Vera or Essential Thrombocythemia

March 11, 2022

Study adds to the evidence that reducing the risk of blood clots should be one of the main goals of managing these myeloproliferative disorders. Monitoring cholesterol levels may be especially important.

New Hemophilia Treatments Look to Cut Number of Infusions

May 12, 2021

Longer half-lives mean less frequent administration and improved quality of life.